Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Case Reports
. 2025 Mar;30(1):e70010.
doi: 10.1111/jns.70010.

Eculizumab as a Disease-Modifying Therapy in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): A Case Report

Edoardo Dalmato Schilke  1   2 , Diletta Cereda  2 , Maria Letizia Fusco  2 , Lorenzo Stanzani  2 , Laura Marzorati  2 , Michela Ripolone  3 , Letizia Bertolasi  3 , Maura Frigo  2 , Franco Molteni  4 , Nico Farina  4 , Carlo Ferrarese  2   5 , Guido Cavaletti  2   6 , Claudia Balducci  2
Affiliations
Case Reports

Eculizumab as a Disease-Modifying Therapy in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): A Case Report

Edoardo Dalmato Schilke et al. J Peripher Nerv Syst. 2025 Mar.

Abstract

Background and aims: Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare immune-mediated disorder; about 20%-30% of patients do not adequately respond to first-line treatments (immunoglobulins, therapeutic plasmapheresis, and corticosteroids) posing diagnostic and therapeutic challenges.

Case report: We report the case of a 58-year-old man diagnosed with CIDP. During follow-up, he progressively became refractory to all first-line treatments. Therefore, 20 months after the diagnosis, an add-on therapy with Rituximab was tried. Despite previous works supporting the use of Rituximab in refractory CIDP, in our case, the patient experienced relapses and progressive increases in disability. After the exclusion of potential CIDP mimics and considering the histological findings that showed complement activation, we opted for an innovative therapeutic approach with Eculizumab that granted a significant clinical and neurophysiological benefit that persists after 7 months of follow-up.

Interpretation: CIDP pathogenesis is characterized by a complex interplay of different immunopathological mechanisms, and the complement system may play a major role. The present case supports the role of complement-dependent toxicity in CIDP and suggests that complement-targeted therapies may represent a well-tolerated and effective alternative for CIDP treatment.

Keywords: chronic inflammatory demyelinating polyneuropathy (CIDP); complement inactivating agents in CIDP; complement system in CIDP; eculizumab in CIDP; refractory CIDP.

PubMed Disclaimer

Figures

FIGURE 1
FIGURE 1
The course of CIDP (evaluated with total ONLS) in our patient with treatments. Time points of electromyography (EMGs) are also reported through the same codes (timepoint a, b, c, d, e, f) used in the article. IVIg, intravenous immunoglobulins; SCIg, subcutaneous immunoglobulins.
FIGURE 2
FIGURE 2
Sural nerve biopsy analyses, light and ultrastructural examination. Moderate reduction in myelinated fibers, with numerous fibers displaying thin myelin sheaths, and asterisks (A). Endoneurial C5b‐9 positive vessel (B). Small onion bulb (C). Macrophage adhering to axon (D). Demyelination in a single‐fiber analysis (E). Bar: (A, B) 25 μm. (C, D) 1 μm.
See this image and copyright information in PMC

References

    1. van den Bergh P. Y. K., van Doorn P. A., Hadden R. D. M., et al., “European Academy of Neurology/Peripheral Nerve Society Guideline on Diagnosis and Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy: Report of a Joint Task Force‐Second Revision,” Journal of the Peripheral Nervous System 26, no. 3 (2021): 242–268, 10.1111/JNS.12455. - DOI - PubMed
    1. Godil J., Barrett M. J., Ensrud E., Chahin N., and Karam C., “Refractory CIDP: Clinical Characteristics, Antibodies and Response to Alternative Treatment,” Journal of the Neurological Sciences 418 (2020): 117098, 10.1016/j.jns.2020.117098. - DOI - PubMed
    1. Querol L. A., Hartung H. P., Lewis R. A., et al., “The Role of the Complement System in Chronic Inflammatory Demyelinating Polyneuropathy: Implications for Complement‐Targeted Therapies,” Neurotherapeutics 19, no. 3 (2022): 864–873, 10.1007/S13311-022-01221-Y. - DOI - PMC - PubMed
    1. Kuwabara S., Kusunoki S., Kuwahara M., et al., “Efficacy and Safety of Eculizumab in Guillain‐Barré Syndrome: A Phase 3, Multicenter, Double‐Blind, Randomized, Placebo‐Controlled Clinical Trial,” Journal of the Peripheral Nervous System 29, no. 3 (2024): 339–349, 10.1111/JNS.12646. - DOI - PubMed
    1. Querol L., Lewis R., Hartung H. P., et al., “Preliminary Efficacy and Safety Data From the Phase 2 Trial of Riliprubart (SAR445088), a Humanized Monoclonal Antibody Targeting Complement C1s, in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (S15.008),” Neurology 102, no. 17_supplement_1 (2024), 10.1212/WNL.0000000000204596. - DOI

Publication types

MeSH terms

LinkOut - more resources